Spinal baclofen pump treatment for children with dystonia
Official title: Intrathecal baclofen and pediatric dystonia.
Funded at Baylor College of Medicine · NIH-11171360 · Data updated 2026-11-12
This project sees whether a spinal baclofen pump can improve movement, reduce pain, and make daily care easier for children with dystonia from cerebral palsy or acquired brain injury.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Baylor College of Medicine NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | September 1, 2024 |
| Project end | August 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $641,262 |
| NIH project number | 5R01HD112563-02 |
| Project ID | NIH-11171360 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children with dystonia related to cerebral palsy or acquired brain injury who are being considered for an intrathecal baclofen pump and can attend serial follow-up visits.
✕Less likely to help
Children whose problems are mainly pure spasticity rather than dystonia, those with medical contraindications to pump implantation, or who cannot access the treatment center may not benefit from this approach.
What this research studies
Children who receive an implanted intrathecal baclofen (ITB) pump will follow a standardized dosing and titration plan and be followed for 12 months. Regular visits will measure dystonia, spasticity, pain, mobility, and caregiving ease using validated assessment tools. Researchers will use clinical exams and brain imaging patterns (for example white matter injury) to compare who improves the most. The study is a prospective observational cohort designed to address limitations of prior small or inconsistent studies.
Why it matters
Potential benefit: If successful, the project could identify which children are most likely to benefit from ITB and lead to better symptom control, less pain, and easier daily care.
How similar studies have gone: Previous studies have shown mixed results for dystonia but some improvement in pain and caregiving, and overall evidence has been limited, so this larger, standardized approach is relatively novel.
Where this research is happening
-
Baylor College of Medicine
Houston, United States Active
Researchers
- Principal investigator: Thomas, Sruthi Pandipati — Baylor College of Medicine
- Study coordinator: Thomas, Sruthi Pandipati
Related research
Other NIH-funded projects on the same topics.
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Role of THAP1 in brain cell support cells shaping brain development
Aims to understand how the THAP1 protein in oligodendrocyte lineage cells helps shape the brain’s environment and myelination during development, which may relate to a movement disorder called DYT6 dystonia.
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Leukocyte RNA biomarkers to help diagnose and monitor dystonia
A small study will look for RNA signatures in blood that could help diagnose and track dystonia in people who have the condition.
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How a dystonia-linked protein affects brain support cells and myelin
This project looks at how the THAP1 protein linked to DYT6 dystonia changes the behavior of brain support cells and the surrounding matrix, which could affect myelination in adults with DYT-THAP1.
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Intrathecal baclofen for children with dystonia due to brain injury
A physician-led study at Baylor College of Medicine will help determine how ITB therapy affects daily life, comfort, and mobility for children with dystonia and related condition after brain injury.
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DYT1 Dystonia: Mouse Models to Understand TorsinA and Brain Circuits
A researcher-led project uses mouse models to explore how a genetic change in DYT1 dystonia affects brain circuits and movement.
-
Dystonia-specific brain signaling pattern in cerebellum
Aims to identify a characteristic pattern of nerve cell activity in the cerebellum that is linked to dystonia, with studies in mice and potential implications for people with the condition.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.